Coalition to Cure Calpain 3 (C3) is excited to announce that a new research grant has been awarded to Dr. Isabelle Richard, Researcher at Généthon, to analyze a novel model of calpainopathy and utilize it for the development of adeno-associated viral (AAV)-mediated gene therapy. This project was funded as a part of the C3 Gene Therapy Initiative, which seeks to accelerate the understanding of gene therapy approaches for calpainopathy.

Dr. Richard is a leader in the field of gene therapy for muscular dystrophies. She was the first to utilize gene therapy to deliver a healthy copy of the calpain 3 gene into a mouse model for calpainopathy. In her current project, "Calpain and animal models: towards therapy," Dr. Richard will characterize a novel model of calpainopathy. She will then use this model to define the dose of calpain 3 gene therapy needed to effectively improve skeletal muscle. This is a crucial step in the development of gene therapy in order to determine the lowest possible dose that is beneficial.

BENEFIT TO PATIENTS

C3 Scientific Director Dr. Jennifer Levy says, "Gene therapy shows incredible promise for treating the muscular dystrophies. Dr. Richard's project will answer central questions about calpain 3 levels needed for restoration of muscle function, which can then be applied to the development of a gene therapy strategy for human patients."